In March and April 2005, CMS issued for comment four draft guidance documents dealing with national coverage determinations. This guidance is required by section 731 of the MMA, which mandates that the Secretary make available to the public the factors CMS considers in making national coverage determinations (NCDs) of whether an item or service is reasonable and necessary. The purpose of these draft guidance documents is to elicit input from interested stakeholders on the factors and processes used by CMS to make NCDs. The three draft guidance documents issued March 9, 2005, discuss the factors CMS considers (1) in opening a national coverage determination, (2) in referring topics to the Medicare coverage Advisory Committee, and (3) in commissioning external technology assessments. In the April 7, 2005, draft guidance document, CMS identifies the factors considered in making a determination of coverage with evidence development. Each draft document had a 60-day comment period. Final guidance documents should be completed within 90 days after the close of the initial 60-day comment periods.

Factors CMS Considers in Opening a National Coverage Determination
This draft guidance describes the initial portion of the NCD process and identifies what CMS considers to be a complete formal request necessary to start the formal review process. Prior to a formal request, the public frequently raises general questions concerning coverage by telephone, mail, or in person. CMS will advise parties on how to request an NCD and offer suggestions to clarify the amount and kind of information necessary, and encourages preliminary meetings.

Formal requests for NCDs can be initiated either by outside parties (a beneficiary, manufacturer, provider, supplier, medical professional association, health plan, or other party) or, rarely, by "aggrieved persons," defined in the statute as "individuals entitled to benefits under Part A or enrolled under Part B or both, who are in need of the item or service that is the subject of the coverage determination." CMS also may generate a request internally to develop an NCD in the interest of general health and safety of Medicare beneficiaries; in situations where providers, patients, or other members of the public have raised significant questions about safety, effectiveness, or obsolescence of items and services, or where interpretation of new evidence or reinterpretation of previously available evidence indicates changes may be warranted; where local coverage policies are inconsistent or conflict with each other to the detriment of beneficiaries; where program integrity concerns have arisen regarding local or national policies; where health technology represents substantial clinical advance and will result in significant health benefits if diffused more rapidly; or where significant uncertainty exists concerning the safety and effectiveness of patient selection or appropriate facility or staffing requirements for new technology.

The guidance sets forth the information necessary for a complete formal request. Should CMS receive a large volume of NCD requests at once, it will prioritize them based on the magnitude of the impact on the Medicare program and beneficiaries.

Factors CMS Considers in Referring Topics to the Medicare Coverage Advisory Committee
This draft guidance sets forth the factors CMS considers in referring issues to the Medicare Coverage Advisory Committee (MCAC), and details the function, structure, process, and role of the MCAC. This referral process is important, because such a referral permits CMS to extend the deadlines. Ordinarily, CMS must release a proposed decision on an NCD within six months after the formal request date. If CMS requests recommendations from the MCAC, that timeframe is nine months.

This guidance document describes the structure of the MCAC. The MCAC is scheduled to hold four to six public meetings each year. Of the maximum of 100 appointed members, no more than 15 members can serve at any one meeting, including a consumer representative and one industry representative. The MCAC gives 30 days notice of a public meeting to discuss the coverage issue. The official transcript and executive summary of the meeting are posted usually within 30 days.

Generally, CMS will refer NCD requests to the MCAC when it needs independent expert advice and assistance in making decisions based upon the "reasoned application of scientific evidence" or "to address broad, significant issues also relevant to coverage policy development," such as the appropriateness of a framework for evaluation of diagnostic tests, assessing lifestyle modifications, interventions to treat cardiac disease, etc. CMS will refer NCD requests to the MCAC in the following circumstances, among others: there is significant controversy among experts regarding the medical benefits of the item or service or the appropriateness of staff or setting, or controversy among the public on the use of the technology; existing published studies contain significant potential methodological flaws, or show conflicting results; and CMS desires more information and/or greater public input including the perspective of potentially affected patients and caregivers.

Factors CMS Considers in Commissioning External Technology Assessments
The Medicare program only covers services that are "reasonable and necessary." In making this determination, CMS must review scientific and clinical evidence available concerning effectiveness of procedures and therapies. CMS typically uses technology assessments (TAs) to assist in the review of evidence in the NCD process. The key component of the TA analysis is the interpretation and critical appraisal of evidence on net health outcomes, after systematic review of the medical literature. This draft guidance sets forth the factors CMS considers in requesting an external TA to supplement the NCD process, including whether the body of evidence to review is extensive and makes completion of an internal TA by CMS questionable within the statutory timeframe; an independent formulation of the appropriate assessment questions and methodology approaches is desirable; significant differences in opinion among experts; review requires unique technical or clinical expertise not available within CMS staff, or specialized methods (e.g., decision modeling); the topic under consideration will be referred to the MCAC; and relevant nonproprietary but unpublished data could be collected and analyzed.

CMS currently contracts with the Agency for Healthcare Research and Quality (AHRQ) for the acquisition of TA reports, and explores with AHRQ the TA feasibility and design. CMS solicited public comments on the role they might play in assisting in the development of questions and the design of the TA while remaining the mandated NCD process timelines.

Factors CMS Considers in Making a Determination of Coverage with Evidence Development
Perhaps the most controversial of the guidance documents is the one describing the factors CMS may consider in a decision to extend coverage for certain items and services while linking such coverage to a requirement for prospective data collection, an approach referred to as "coverage with evidence development" (CED). CMS does not anticipate a substantial number of new CEDs, but will continue to approve CEDs in specific cases where better evidence to support decision making by patients and clinicians is an essential part of reaching a conclusion that treatment is reasonable and necessary.

This linking of coverage with future evidence development is intended to enable Medicare to provide payment for items and services under conditions that help assure significant net benefits for the treatment of beneficiaries. Additional studies may help target those patients who benefit most in a broad population, or identify additional patients who benefit beyond a narrowly studied population; or may include information on side effects or complications of treatments. Such data collection should continue only as long as important questions remain. The CED process permits coverage to be granted earlier than it might be if CMS waited until all potential questions were answered.

Generally, CEDs will be issued in two circumstances:

A particular medical intervention may have been demonstrated to improve health outcomes for a broad population of patients, but the evidence would only be adequate, and the service therefore reasonable and necessary for the individual patient, when the provider collects and reviews specific data at the time the service is delivered. An example of this is the recent NCD extending coverage of implantable cardioverter defibrillators (ICDs) to a broader array of patients, combined with a registry to record information on those patients.
When a medical intervention has yet to conclusively demonstrate an improvement in health outcomes but existing information suggests the intervention may provide a benefit. An example is the coverage of anti-cancer drugs approved for colorectal cancer in specific NCI-sponsored clinical trials.

CMS will consider requiring CEDs where there are still substantial safety concerns or potential side effects that are inadequately described; where risks and benefits for off-label use have not been adequately addressed in the literature; where available clinical studies may not have adequately described risks and benefits in specific patient subgroups; and where comprehensive evidence of effectiveness for treatments of rare diseases is not always available or feasible to develop in a pre-market setting.